ToolGen to enter Phase 1 trial of gene-editing therapy for ovarian cancer by early 2023

South Korean CRISPR gene-editing company ToolGen Inc. aims to start a Phase 1 clinical trial of its CAR-T platform to treat ovarian cancer as early as December or by the beginning of next year.

The new therapy named Styx-T under development jointly with an Australian biotech firm is at the most advanced stage of clinical development among ToolGen’s pipeline. This treatment requires culturing immune cells corrected with a gene editing tool in vitro and then injecting them into the patient’s bone marrow. A preclinical study is being conducted in Australia.

The Korean biotech company Thursday said it is considering several options, including an overseas location for the first phase study, which will begin in December of this year at the earliest or early next year at the latest.

ToolGen is also speeding up on other clinical development programs based on its patented genome editing technology. It plans to submit an Investigational New Drug (IND) application for its therapy targeting Charcot Marie Tooth (CMT) 1A in 2023. CMT is a hereditary peripheral neuropathy with a relatively large number of patients, with an incidence of 1 in 2,500 people, but there is no cure in the world. The company’s novel drug for wet macular degeneration is being tested in primates, and it aims to start a Phase 1 clinical trial in the United States next year.

ToolGen headquartered in Seoul’s Gasan Digital Complex is a pioneer in the use of the “genetic scissors” for the development of therapies to target gene-related rare diseases. ToolGen is the only Korean company that has CRISPR technology, and globally it has only two rivals: UC Berkeley and Broad Institute of MIT and Harvard. ToolGen is confronting with the top U.S. schools in patent interference dispute over the CRISPR technology, but the company is confident about its triumph.

According to market data, the global gene editing market is growing at a rapid pace from $1.23 billion in 2020 to $2.3 billion in 2022.

Kosdaq-listed ToolGen shares added 0.61 percent to 66,400 won ($50.05) during mid-day session on Friday.

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